Congenital Heart Disease Clinical Trial
Official title:
Use of Oxandrolone to Promote Growth in Infants With Hypoplastic Left Heart Syndrome: A Phase I/II Pilot Study
Verified date | July 2023 |
Source | Carelon Research |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The primary aim of this study is to determine if clinically relevant doses of buccally administered oxandrolone are safe and tolerable in neonates with hypoplastic left heart syndrome (HLHS) or other single right ventricular anomalies who have undergone a Norwood procedure. The secondary aim is to evaluate the efficacy of buccally administered oxandrolone in improving objective indices of growth and nutrition in neonates who have undergone a Norwood procedure.
Status | Terminated |
Enrollment | 34 |
Est. completion date | September 19, 2023 |
Est. primary completion date | September 19, 2023 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 14 Days |
Eligibility | Inclusion Criteria: 1. HLHS and other single ventricle of right ventricular morphology 2. Age and Norwood procedure =14 days of age 3. Informed consent from parent/guardian Exclusion Criteria: 1. Small for gestational age (birth weight <10th percentile for gestational age) 2. Prematurity, defined as gestational age <37 weeks 3. Intrauterine growth retardation (birth weight =2.5 kg and gestational age =38 weeks) 4. Chromosomal abnormality, recognizable genetic syndrome or congenital anomalies of more than minor severity associated with growth failure 5. Moderate or greater right ventricular systolic dysfunction and/or moderate or greater tricuspid regurgitation prior to the Norwood procedure 6. Extracorporeal membrane oxygenation support (ECMO) prior to or within 24 hours of Norwood procedure 7. Pre-Norwood interventions (fetal intervention, balloon atrial septostomy for an intact or restrictive atrial septum) 8. Pre-Norwood pulmonary venous obstruction 9. Pre-Norwood procedure necrotizing enterocolitis and/or other gastrointestinal syndromes 10. Known contraindication to oxandrolone 11. Planned or current warfarin therapy at screening (warfarin effects are increased by anabolic drugs) 12. Significant hepatic dysfunction (elevation of serum transaminase levels greater than two times the upper limit of normal local laboratory standard at screening) 13. Hypercalcemia (>1.5 times upper normal range for lab) 14. Nephrotic syndrome 15. Unwillingness or inability to return to surgical center for follow-up evaluation 16. Participation in another clinical study that may impact growth |
Country | Name | City | State |
---|---|---|---|
Canada | The Hospital for Sick Children | Toronto | Ontario |
United States | University of Michigan Health System, Ann Arbor | Ann Arbor | Michigan |
United States | Children's Hospital of Atlanta | Atlanta | Georgia |
United States | Boston Children's Hospital | Boston | Massachusetts |
United States | Medical University of South Carolina | Charleston | South Carolina |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | Cook Children's Medical Center | Fort Worth | Texas |
United States | Texas Children's Hospital | Houston | Texas |
United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | Primary Children's Medical Center | Salt Lake City | Utah |
Lead Sponsor | Collaborator |
---|---|
Carelon Research |
United States, Canada,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Biochemical evidence of hepatic dysfunction | Elevation of serum transaminase levels (alanine transaminase (ALT) and/or aspartate transaminase (AST)) >4 times the local laboratory upper limit of normal | From date of treatment initiation until the pre-SCPC evaluation or end of study participation, whichever comes first, up to 9 months | |
Primary | Virilization | Standardized physical examination will be performed. Because there are no standard normal values for the various measurements included, each subject will serve as their own control | From date of treatment initiation until the completion of study drug therapy or end of study participation, whichever comes first, up to 28 days | |
Primary | SAE probably or definitely related to oxandrolone therapy | Any SAE probably or definitely related to oxandrolone therapy in the opinion of the medical monitor | From date of treatment initiation until the pre-SCPC evaluation or end of study participation, whichever comes first, up to 9 months | |
Secondary | Length-for-age z-score | The efficacy of buccally administered oxandrolone will be evaluated by measuring length-for-age z-score at the end of study drug therapy | At the time of completion of study drug therapy, up to 28 days after date of treatment initiation | |
Secondary | Weight-for-age z-score | The efficacy of buccally administered oxandrolone will be evaluated by measuring weight-for-age z-score at the end of study drug therapy | At the time of completion of study drug therapy, up to 28 days after date of treatment initiation | |
Secondary | Change in Weight-for-age z-score | The efficacy of buccally administered oxandrolone will be evaluated by measuring the change in weight-for-age z-score at the end of study drug therapy | From date of pre-Norwood procedure until completion of study drug therapy, up to 28 days | |
Secondary | Change in length-for-age z-score | The efficacy of buccally administered oxandrolone will be evaluated by measuring the change in length-for-age z-score at the end of study drug therapy | From date of pre-Norwood procedure until completion of study drug therapy, up to 28 days | |
Secondary | Prealbumin levels | Serum prealbumin levels will be measured weekly | During the duration of therapy | |
Secondary | Lean Body Mass | Lean body mass will be assessed using dual energy x-ray absorptiometry (DXA) | At the completion of study drug therapy, assessed up to 35 days after initiation of study drug therapy | |
Secondary | Decreased right ventricular systolic function | Evidence of =moderate right ventricular systolic dysfunction or tricuspid valve regurgitation based on qualitative assessment of clinical echocardiograms if performed | At the time of Norwood discharge and at the time of pre-SCPC evaluation, up to 9 months |
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